Phase 1 Study to Assess Safety and Efficacy of ANG003

Anagram Therapeutics, Inc.51 enrolled

Overview

Randomized, parallel, active-treatment Phase 1 study of a single dose of orally administered ANG003 with a test meal in adult subjects with cystic fibrosis-related exocrine pancreatic insufficiency. The study's overall objectives are to evaluate the safety, tolerability and effect of four dose levels of ANG003.

The Phase 1 study was designed to compare a 24h Baseline Substrate Absorption Challenge Test (SACT) period with no enzymes to a 24h ANG003 SACT period with enzymes. Eligible subjects will be randomly assigned with equal allocation to one of four active dose levels of lipase, protease and amylase. Approximately 48 to 60 eligible subjects are planned to be enrolled in the study with 12 to 15 subjects assigned to each dose level from up to 21 investigational sites.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Exocrine Pancreatic Insufficiency

Interventions

ANG003DRUG

To evaluate four possible combinations of lipase, protease and amylase.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Male and female subjects 18 years of age or older. 2. Confirmed diagnosis of CF defined as: a) CF signs and symptoms AND b) Two CF-causing mutations on genetic testing or sweat chloride \>60 mEq/L. 3. Documented history of fecal elastase \<100 µg/g stool. 4. EPI clinically controlled with minimal clinical symptoms and on a stable dose of PERT for 90 days before screening as determined by the Investigator. 5. Adequate nutritional status measured by body mass index ≥20kg/m2 for adult subjects. Exclusion Criteria: 1. Subjects with diabetes mellitus who are unable to refrain from short-acting and rapid-acting insulin on Days 1 and 5 for a daily total of 6 hours. 2. Involuntary loss of ≥10% of usual body weight within last 6 months or involuntary loss of \>5% of body weight within 1 month. 3. Requires use of naso-gastric, J-tube, G-tube, and/or enteral feeding for the study duration. 4. CF pulmonary exacerbation within 30 days prior to the Baseline SACT Period (Visit 2). 5. Subjects who cannot discontinue omega-3 supplements \>500 mg of DHA and EPA daily. 6. Subjects unable to tolerate missing a dose of PERT.

Locations (20)

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Outcomes

Primary Outcomes

Adverse Events (AE), Serious Adverse Events (SAE), and AEs Leading to Study Discontinuation

AE, SAE, and AEs leading to study discontinuation measured by frequency and severity during study.

Time frame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).

Malabsorption Symptoms

Severity of malabsorption symptoms (e.g., abdominal discomfort or pain, bloating, heartburn, regurgitation or reflux, retching, stomach fullness, and vomiting) by patient reported outcome scale (0=None; 1=Very Mild; 2=Mild; 3=Moderate; 4=Severe; 5=Very Severe). Measured by 20 questions contained in Acute Patient Assessment of Gastrointestinal Symptoms (Acute PAGI-SYM) questionnaire.

Time frame: Acute PAGI-SYM is based upon 7-day recall.

Secondary Outcomes

Fat Absorption

Measured by concentration and percent of plasma fatty acids.

Time frame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).

Protein Absorption

Measured by changes in plasma concentration of amino acids.

Time frame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).

Carbohydrate Absorption

Changes in glucose (mg/dL) as measured by continuous glucose monitoring.

Time frame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).

Data from ClinicalTrials.gov

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